Multiple SclerosisPhase 1/2NCT00278655
Hematopoietic Stem Cell Therapy for Patients With Multiple Sclerosis
A Phase 1/2 study testing Hematopoietic stem cell transplantation in people with Multiple Sclerosis, run by Northwestern University.
Trial summary
Multiple sclerosis is disease believed to be due to immune cells, cells which normally protect the body, but are now attacking the tissue in the brain and possibly the spinal cord. The likelihood of progression of this disease is high. Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Multiple Sclerosis
- What is being tested?
- Hematopoietic stem cell transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- No sites listed yet
- Study dates
- Jun 2003to May 2012
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age between 18-50, inclusive.
- Diagnosis of Multiple Sclerosis (MS) using Poser criteria (Appendix A).
- An Expanded Disability Status Scale (EDSS) of 2.0 - 5.5 (Appendix B).
- Inflammatory disease despite primary disease modifying therapy with at least 3 months of interferon. Failure is defined as two or more clinical relapses with documented neurologic changes within the year prior to the study. (NOTE: Relapses must have required treatment with corticosteroids. Sensory only relapses are excluded.) Failure may also be defined as one relapse within the year prior to study if there is evidence on MRI of active inflammation (i.e., gadolinium enhancement).
Who cannot join
- Any illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive chemotherapy.
- Prior history of malignancy except localized basal cell, squamous skin cancer or carcinoma in situ of the cervix. Other malignancies for which the patient is judged to be cured, such as head and neck cancer, or breast cancer will be considered on an individual basis.
- Positive pregnancy test.
- Inability or unwillingness to pursue effective means of birth control. Effective birth control is defined as 1) refraining from all acts of vaginal intercourse (ABSTINENCE); 2) consistent use of birth control pills; 3) injectable birth control methods (Depo-provera, Norplant); 4) tubal sterilization or male partner who has undergone vasectomy; 5) placement of an intrauterine device (IUD); or 6) use, with every act of intercourse, of diaphragm with contraceptive jelly and/or condoms with contraceptive foam.
- Failure to willingly accept or comprehend irreversible sterility as a side effect of therapy.
- Forced expiratory volume in 1 second (FEV1) / forced vital capacity (FVC) < 60% of predicted after bronchodilator therapy (if necessary).
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- Diffusing capacity of the lung for carbon monoxide (DLCO) < 50% of predicted.
- Resting left ventricular ejection fraction (LVEF) < 50 %.
- Bilirubin > 2.0 mg/dl.
- Serum creatinine > 2.0 mg/dl.
- Known hypersensitivity to mouse, rabbit, or E. Coli derived proteins, or to iron compounds/medications.
- Presence of metallic objects implanted in the body that would preclude the ability of the patient to safely have MRI exams.
- Diagnosis of primary progressive multipole sclerosis (MS).
- Platelet count < 100,000/ul.
- Psychiatric illness, mental deficiency or cognitive dysfunction making compliance with treatment or informed consent impossible.
- Active infection except asymptomatic bacteruria.
About this study
Official title: Hematopoietic Stem Cell Therapy for Patients With Inflammatory Multiple Sclerosis Failing Interferon Therapy: A Phase II Multi-Center Trial
Multiple sclerosis is disease believed to be due to immune cells, cells which normally protect the body, but are now attacking the tissue in the brain and possibly the spinal cord. The likelihood of progression of this disease is high. This study is designed to examine whether treating patients with high dose cyclophosphamide and CAMPATH-1H (drugs which reduce the function of the immune system) followed by return of previously collected blood stem cells will stop the progression of your multiple sclerosis. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the cyclophosphamide and CAMPATH-1H is to destroy the cells in your immune system which are thought to be causing your disease. The purpose of the stem cell infusion is to restore the body's blood production, which will be severely impaired by the high dose chemotherapy and to produce a normal immune system that will no longer attack the body.
- Conditions
- Multiple Sclerosis
- Being tested
- Hematopoietic stem cell transplantation
- Medical subject headings
- Multiple Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hematopoietic stem cell transplantationBiological
Autologous hematopoietic stem cell transplantation
| Study group | Role | What they receive |
|---|---|---|
| Hematopoietic stem cell transplantation | Experimental | All participants will undergo hematopoietic stem cell transplantation after receiving conditioning regimen. Receives: Biological: Hematopoietic stem cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease Progression
Data are reporting number of participants with disease progression. Disease progression is defined as a 1 point increase in the Expanded Disability Status Scale (EDSS) on consecutive evaluations at least 3 months apart.
Measured: 3 years after transplant
Secondary outcomes (1)
Survival
Data are reporting the number of participants who survived three years after the transplant
Survival of 21 participants was evaluated at three years after the transplant
Measured: three years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2006 (estimated)
- Study startJun 2003
- Primary completionDec 2011
- Study completionMay 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Multiple SclerosisStudies of the same condition, open and under way.
- More trials run by Northwestern UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northwestern University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Richard Burt, MD, Northwestern University
- Investigators
- Richard Burt, MD, Principal investigator, Northwestern University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00278655
- Sponsor's protocol ID
- DIAD MS.Auto2002
- Status last verified
- Mar 2014
- First submitted
- 16 Jan 2006
- Last updated
- 1 May 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hematopoietic Stem Cell Therapy for Patients With Inflammatory Multiple Sclerosis Failing Interferon Therapy: A Phase II Multi-Center Trial
- ClinicalTrials.gov ID
- NCT00278655
- Acronym / sponsor ID
- DIAD MS.Auto2002
- Conditions
- Multiple Sclerosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Northwestern UniversityUniversity, hospital or other
- Study start
- Jun 2003
- Primary completion
- Dec 2011 (actual)
- Study completion
- May 2012 (actual)
- First posted
- 18 Jan 2006 (estimated)
- Results
- Posted 1 May 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Multiple sclerosis is disease believed to be due to immune cells, cells which normally protect the body, but are now attacking the tissue in the brain and possibly the spinal cord. The likelihood of progression of this disease is high. This study is designed to examine whether treating patients with high dose cyclophosphamide and CAMPATH-1H (drugs which reduce the function of the immune system) followed by return of previously collected blood stem cells will stop the progression of your multiple sclerosis. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the cyclophosphamide and CAMPATH-1H is to destroy the cells in your immune system which are thought to be causing your disease. The purpose of the stem cell infusion is to restore the body's blood production, which will be severely impaired by the high dose chemotherapy and to produce a normal immune system that will no longer attack the body.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hematopoietic stem cell transplantation | Experimental | Biological: Hematopoietic stem cell transplantationAll participants will undergo hematopoietic stem cell transplantation after receiving conditioning regimen. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Age between 18-50, inclusive.
- Diagnosis of Multiple Sclerosis (MS) using Poser criteria (Appendix A).
- An Expanded Disability Status Scale (EDSS) of 2.0 - 5.5 (Appendix B).
- Inflammatory disease despite primary disease modifying therapy with at least 3 months of interferon. Failure is defined as two or more clinical relapses with documented neurologic changes within the year prior to the study. (NOTE: Relapses must have required treatment with corticosteroids. Sensory only relapses are excluded.) Failure may also be defined as one relapse within the year prior to study if there is evidence on MRI of active inflammation (i.e., gadolinium enhancement).
Key exclusion criteria (16 listed)
- Any illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive chemotherapy.
- Prior history of malignancy except localized basal cell, squamous skin cancer or carcinoma in situ of the cervix. Other malignancies for which the patient is judged to be cured, such as head and neck cancer, or breast cancer will be considered on an individual basis.
- Positive pregnancy test.
- Inability or unwillingness to pursue effective means of birth control. Effective birth control is defined as 1) refraining from all acts of vaginal intercourse (ABSTINENCE); 2) consistent use of birth control pills; 3) injectable birth control methods (Depo-provera, Norplant); 4) tubal sterilization or male partner who has undergone vasectomy; 5) placement of an intrauterine device (IUD); or 6) use, with every act of intercourse, of diaphragm with contraceptive jelly and/or condoms with contraceptive foam.
- Failure to willingly accept or comprehend irreversible sterility as a side effect of therapy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Hematopoietic stem cell transplantationAutologous hematopoietic stem cell transplantation | Biological | Hematopoietic stem cell transplantation |
Primary outcome
Disease Progression
Time frame: 3 years after transplant
Data are reporting number of participants with disease progression. Disease progression is defined as a 1 point increase in the Expanded Disability Status Scale (EDSS) on consecutive evaluations at least 3 months apart.
1 secondary outcome
Survival
Time frame: three years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 May 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Multiple Sclerosis
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Non-interventional Study of Subcutaneous Ocrelizumab in Persons With Relapsing-Remitting Multiple Sclerosis
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A Phase 2a Study of SUN-627 in Participants With Non-Active Secondary Progressive and Primary Progressive Multiple Sclerosis (SPMS/PPMS)
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A Study of Ocrelizumab Administered Subcutaneously in Participants With Multiple Sclerosis Who Switch From an Approved Anti-CD20 Therapy
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Richard Burt, MD principal investigator
Northwestern University
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Hematopoietic stem cell transplantation in people with Multiple Sclerosis, run by Northwestern University.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 1 May 2014. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 May 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Disease Progression (time frame: 3 years after transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 May 2014.